CompletedPhase 1

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy

United States, Canada59 participantsStarted 2021-02-16

Plain-language summary

This is a phase 1, multi-center, double-blind, placebo-controlled, multiple dose escalation study with NIO752 in progressive supranuclear palsy (PSP) participants.

Who can participate

Age range40 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Signed informed consent
✓. Between 40 to 75 years old (inclusive)
✓. Have PSP diagnosed for less than 5 years with a current classification of probable PSP Richardson syndrome, a progressive supranuclear palsy rating scale (PSPRS) score \< 40 and MOCA score \>17 at screening
✓. Be able to ambulate independently or able to take at least 5 steps with minimal assistance
✓. At least a 12-month history of postural instability or falls within 3 years from disease onset as per medical history
✓. Vertical supranuclear gaze palsy, or reduced velocity of vertical saccade
✓. Able and willing to meet all study requirements including:
✓. If the participant is receiving levodopa/carbidopa, levodopa/benserazide, a dopamine agonist, catechol-o-methyltransferase (COMT) inhibitor, rasagiline, CoQ10 or other Parkinson's medications, acetylcholinesterase inhibitors, antipsychotics, memantine, or other non-tau modifying Alzheimer's medication the dose must have been stable for at least 30 days prior to the screening visit and must remain stable for the duration of the study. No such medication can be initiated during the study.

Exclusion criteria

✕. Live in a skilled nursing facility or dementia care facility
✕. Evidence of motor neuron disease, or any other neurological disease that could explain symptoms
✕. Clinically significant laboratory abnormality
✕. Attempted suicide, suicidal ideation with a plan that required hospital admission within 12 months prior to Screening. In addition, patients deemed by the Investigator to be at significant risk of suicide, major depressive episode, psychosis, confusion state, or violent behavior should be excluded.
✕. A clear and robust benefit from levodopa by history
✕. Use of lithium, methylene blue or other putative disease modifying drugs for PSP within 30 days of screening
✕. Any previous use of experimental therapy within 30 days or 5 half-lives prior to Day 1, whichever is greater
✕. Any condition that increases risk of meningitis unless participant is receiving appropriate prophylactic treatment

What they're measuring

Number of adverse events and serious adverse events

Timeframe: Baseline up to approximately one year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

Change in severity scores for Columbia-Suicide Severity Rating Scale (C-SSRS)

Timeframe: Baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

Levels of infection indicators in Cerebrospinal fluid (CSF)

Timeframe: Baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

Trial details

NCT IDNCT04539041

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-10-17

View on ClinicalTrials.gov More Progressive Supranuclear Palsy (PSP) trials

Who can participate

Age range40 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Signed informed consent
✓. Between 40 to 75 years old (inclusive)
✓. Have PSP diagnosed for less than 5 years with a current classification of probable PSP Richardson syndrome, a progressive supranuclear palsy rating scale (PSPRS) score \< 40 and MOCA score \>17 at screening
✓. Be able to ambulate independently or able to take at least 5 steps with minimal assistance
✓. At least a 12-month history of postural instability or falls within 3 years from disease onset as per medical history
✓. Vertical supranuclear gaze palsy, or reduced velocity of vertical saccade
✓. Able and willing to meet all study requirements including:
✓. If the participant is receiving levodopa/carbidopa, levodopa/benserazide, a dopamine agonist, catechol-o-methyltransferase (COMT) inhibitor, rasagiline, CoQ10 or other Parkinson's medications, acetylcholinesterase inhibitors, antipsychotics, memantine, or other non-tau modifying Alzheimer's medication the dose must have been stable for at least 30 days prior to the screening visit and must remain stable for the duration of the study. No such medication can be initiated during the study.

Exclusion criteria

✕. Live in a skilled nursing facility or dementia care facility
✕. Evidence of motor neuron disease, or any other neurological disease that could explain symptoms
✕. Clinically significant laboratory abnormality
✕. Attempted suicide, suicidal ideation with a plan that required hospital admission within 12 months prior to Screening. In addition, patients deemed by the Investigator to be at significant risk of suicide, major depressive episode, psychosis, confusion state, or violent behavior should be excluded.
✕. A clear and robust benefit from levodopa by history
✕. Use of lithium, methylene blue or other putative disease modifying drugs for PSP within 30 days of screening
✕. Any previous use of experimental therapy within 30 days or 5 half-lives prior to Day 1, whichever is greater
✕. Any condition that increases risk of meningitis unless participant is receiving appropriate prophylactic treatment

What they're measuring

Number of adverse events and serious adverse events

Timeframe: Baseline up to approximately one year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

Change in severity scores for Columbia-Suicide Severity Rating Scale (C-SSRS)

Timeframe: Baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

Levels of infection indicators in Cerebrospinal fluid (CSF)

Timeframe: Baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)